Urge the FDA to Say YES to Accelerated Approval For Therapies For Duchenne.

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We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy – the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United […]

Emmaus Medical Announces Completion of All Patient Visits For its Phase 3 Clinical Trial for Treatment of Sickle Cell Disease


Emmaus Medical, Inc., a biopharmaceutical company engaged in the discovery, development and commercialization of innovative and cost-effective treatments and therapies primarily for rare and orphan diseases, today announced it has completed all patient visits for its Phase 3 clinical trial for the treatment of sickle […]

Chelsea’s Northera Gets Another Chance at Treating Autonomic Dysfunction

Chelsea's Northera

Investors betting Chelsea would win FDA approval for its drug Northera for neurogenic orthostatic hypotension, or NOH, in multiple systems atrophy and Parkinson’s patients, were dealt a disappointing blow when the FDA opted against approving the drug in 2012. However, the FDA’s willingness to consider data from […]

“I Had My DNA Picture Taken, With Varying Results”- New York Times


Kira Peikoff, 28, writes for the New York Times on her experience with DNA picturing. I like to plan ahead; that much I knew about myself before I plunged into exploring my genetic code. I’m a healthy 28-year-old woman, but some nasty diseases run in […]

Brothers with Morquio syndrome to Try Experimental Treatment in Toronto


Two brothers from the Wingham area, diagnosed with a rare disease, are hoping to add some years to their lives. The teenagers, Zane and Luke Braun, travel to Toronto every two weeks– and have been doing so for the past two years– for an experimental […]

Keck Medicine Physicians First to Implant Epilepsy-Controlling Device


Keck Medicine of USC has become the world’s first medical center to implant a responsive brain device newly approved by the Food and Drug Administration (FDA) to treat epilepsy. It has the potential to help millions of people worldwide. The treatment involves surgical implantation of […]

FDA Approves Tretten to Treat Rare Genetic Clotting Disorder


The U.S. Food and Drug Administration today approved Tretten, Coagulation Factor XIII A-Subunit (Recombinant), the first recombinant product for use in the routine prevention of bleeding in adults and children who have a rare clotting disorder, known as congenital Factor XIII A-subunit deficiency. Congenital Factor […]